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- Title
Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM).
- Authors
Brod, Meryl; Bushnell, Donald M.; Neergaard, Jesper Skov; Waldman, Laura Tesler; Busk, Anne Kirstine
- Abstract
Background: To capture the broad range of treatment burden issues experienced by adolescent and adult people with hemophilia (PWH), the Hemophilia Treatment Experience Measure (Hemo-TEM) was developed. We describe the development of this new hemophilia-specific patient-reported outcome (PRO) measure including concept elicitation, cognitive debriefing, and psychometric validation. Results: Concept elicitation interviews were conducted with 5 clinical experts and 30 adult PWH in the United States (US). The qualitative analysis of these interviews and a review of the literature informed the PRO measure development. The project team reviewed concept endorsement rates and generated a 27-item preliminary version of the Hemo-TEM. Cognitive debriefing interviews were conducted to ensure participant understanding and item relevance in samples of (adolescent (n = 20) and adult (n = 14)) PWH in the US. The refined, validation-ready version of the Hemo-TEM included 30 items. Lastly, data from 3 clinical trials comprised the 4 analysis sets used for the psychometric validation with a sample size of N = 88. Item reduction dropped 4 items resulting in a final 26-item measure. Factor analysis generated 5 domains in the Hemo-TEM [injection difficulties (3 items), physical impact (6 items), treatment bother (7 items), interference with daily life (4 items), and emotional impact (6 items)] and a total score. All scores were reliable [internally consistent (0.84–0.88)]. For convergent validity, with the exception of one domain, all hypothesized associations were met. Preliminary sensitivity to change effect sizes were between − 0.30 and − 0.70. Meaningful change thresholds ranged from 6 points (physical impact and emotional impact) to 10 points (treatment bother) with 8 points for the Hemo-TEM total score. Conclusions: Findings from the concept elicitation, cognitive debriefing, and psychometric validation phases provide evidence that the Hemo-TEM is a well-designed, valid, and reliable measure of the burden of hemophilia treatment, including treatment impact on adolescent and adult PWH.
- Subjects
UNITED States; HEMOPHILIA treatment; EXPERIMENTAL design; THERAPEUTICS; RESEARCH evaluation; RESEARCH methodology evaluation; RESEARCH methodology; ATTITUDE (Psychology); HEALTH outcome assessment; INTERVIEWING; PATIENTS' attitudes; PSYCHOMETRICS; QUALITATIVE research; QUESTIONNAIRES; FACTOR analysis; RESEARCH funding
- Publication
Journal of Patient-Reported Outcomes, 2023, Vol 7, Issue 1, p1
- ISSN
2509-8020
- Publication type
Article
- DOI
10.1186/s41687-023-00550-6